Potential treatments for rare genetic diseases have promise; ‘we need to be ready’

[caption id="attachment_29726" align="alignright" width="360" caption="BioMarin completed a $60 million-plus capital project last year to more than double commercial production capacity at the eight-building campus in the Bel Marin Keys industrial area."][/caption]

NOVATO -- Construction crews have been busy preparing BioMarin Pharmaceutical's facilities in the city for a doubling of production to deliver treatments for testing and sale as well as hiring at least a couple of hundred more employees.

BioMarin completed a $60 million-plus capital project last year to more than double commercial production capacity at the eight-building campus in the Bel Marin Keys industrial area. This year the company continues to expand administrative departments to a ninth building, is upgrading utilities and laboratories and planning further manufacturing growth in 2012, according to Robert Cone, director of capital projects and facilities engineering.

One hundred employees joined the company last year, bringing the total to 700. Roughly another 100 local hires are planned for this year.

"It all depends on the success of our clinical programs," he said. "We need to be ready."

The publicly traded company has grown considerably from its 1997 launch with $1.5 million in funding to 2010 revenue estimated at $370 million to $393 million. Currently in commercial production are Naglazyme, Kuvan, Aldurazyme and Firdapse treatments for rare genetic diseases. Firdapse currently is approved for sale only in Europe.

In addition to commercial production, BioMarin needs to make testing amounts for several treatments in various stages of trials. For example, in the first half of this year, BioMarin will be producing phase 3 clinical test samples of the GALNS enzyme-replacement treatment, called product BMN 110, for Morquio A syndrome, as well as doses of Firdapse for phase 3 trials in the U.S.

Juggling commercial and testing production called for upgrades to the three manufacturing buildings. It's an involved process for products under close government scrutiny.

The production line for the Naglazyme treatment, BioMarin's largest revenue-generator, was moved from one portion of a building to another to increase capacity between early 2009 and early 2010.

The rest of the year was taken up with "process qualification," Consultants must verify the quality of water, air and other environmental factors meet U.S., European and other regulatory standards during full-scale production.

"We are taking commercial manufacturing space and making clinical products and then flipping it back to commercial production," Mr. Cone said.

BioMarin has benefited from the economic recession because it has had access to equipment that would have been bought up by large pharmaceutical manufacturers, he said.

Upgrading the 46 Galli Drive facility, called Galli East, had to be done without affecting commercial and clinical production in adjacent facilities with dust and vibrations. So the building was completed from the inside out, with support piers drilled rather than pounded inside the existing building before the shell was demolished. That allowed work to continue during the winter.

During the process qualification phase of the project last year and continuing into this year, Novo Construction of San Francisco has been upgrading aging Bel Marin Keys utilities to handle BioMarin's current and anticipated production.

That includes installation of wastewater pretreatment units in the three production buildings. Novo has been working at the plant consistently since 2005, including direction of major shutdown maintenance in December.

Meanwhile, the company is expanding into a ninth building, moving administrative departments to Novato office space located about 10 minutes away, to accommodate a growing headquarters workforce.

"As we get bigger, the floorplates around here do not accommodate the adjacencies we need," Mr. Cone said.

BioMarin is upgrading local employment opportunities and diversifying the local economy, according to Ron Gerber, city redevelopment and economic development director.

"BioMarin is a great economic and employment asset," Mr. Gerber said.

The company has created a number of well-paying jobs and is changing the Bel Marin Keys business park from light-industrial uses such as catering kitchens and windshield installers to office-using businesses such as technology companies.

BioMarin itself has helped form a biotech cluster of innovation companies in the city together with The Buck Institute for Age Research, Mr. Gerber noted. [See "Buck, BioMarin in joint research effort," Sept. 20.] Another example is Raptor Pharmaceutical, formed in 2009 by former BioMarin employees.